Novel Genomic Screening Tool Enables Precision Reverse-Engineering of Genetic Programming in Cells

Collaborative Research Led by Investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Defines A Novel Approach to Understanding How Called Transcript Factors Determine Determs determine program program Drive Cell Growth and Maturation. The Study is published In the Journal Science.

The Method, Called “Disturbing-Multiome,” Uses Crispr to Knock Out the Function of Individual Transcription Factors Across Many Blood Cells at Once. The Researchers then Perform Single-Cell Analyses on Each Cell to Measure the Effects of the Editing, Including Identifying Which Genes have Been Turned on Oro and Which Genes Are Accessible (Based on Epigenetic Markers). The Team Appled This TOOL TO IMMMATURE BLAOD CELLS TO IDENTIFY THE IMPORTANT TRANSITRATION FATORS – AOD DNA REGIONS THAT CODE FOR THEM – THAT STRONGLY AFFECT How Blood Cells Develop.

They Discoved That Many of the DNA Regional Thes identified as Important for Driving Blood Cell Production are also regions Known to harbor mutations Linked to Blood Disorders. The DNA regional they identified as Important Occupy Less Than 0.3% of the Genome, Buty Explain A Disprortionity Large Share of the Genetic Influence on Blood Cell Features and Specialization.

In Earlier Work, Investigators from this Team and other collaborators used Genome-Wide Association Studies to Identify the Transcription Responsible for Switching Off Fetal Hemoglobin After Birth, Laying The Groundwork for the Development of Gene Therapy for Sickle Cell Disease And Beta Thalassemia.

This New Disturbing Approach Enables Researchers to Systematically Reveal How Thoughs of Transcription Factor Variants Influence Blood Production and Influence Disease Risk, Creatation Opportunities for Finding Many More Novel Targeted Therapies for Blood Disorders.